26 posts from February 2015

February 27, 2015

According to America Speaks, a compilation of public opinion polls commissioned by Research!America, the majority of Americans agree with the central tenets of the 21st Century Cures Bill.

"Majorities across the political spectrum say it is important that the new 114th Congress takes action on assuring the discovery, development and delivery of treatments and cures for diseases in the first 100 days of the legislative session (75% Democrats, 64% Republicans and 60% Independents)," states Research!America. "As Congress considers numerous proposals in support of research, including the 21st Century Cures draft legislation aimed at speeding the delivery of lifesaving treatments to patients, it is notable to see public support in favor of accelerating medical progress."

The report found that an increasing percentage of Americans say the U.S. Food and Drug Administration (FDA) should move more quickly in order to get new treatments to patients, even if it means there may be risks. In 2015, 38% favor faster regulatory review, compared to 30% in 2013 (see the graphic to the left). Meanwhile, 25% say the FDA should act more slowly in order to reduce risk, even if it means patients may wait longer for treatments. Another 19% are undecided on this question and 18% do not agree with either position.

When it comes to rising health care costs, 46% say research to improve health is part of the solution, while 28% are not sure and 26% say research is part of the problem. Meanwhile, 41% say that the roughly 1.5% of government spending allocated for biomedical and health research is not enough. Nearly one-third (29%) say it is about right, 21% are not sure and 9% say it is too much.

Furthermore, 44% say they are willing to pay $1 per week more in taxes if they were certain that all of the money would be spent on additional medical research, while 32% say no and 24% are not sure.

Currently, only 27% of Americans believe the U.S. has the best health care system in the world, but more than half say it is important that the U.S. is a leader in medical and health research. Furthermore, confidence in the current system in the U.S. for evaluating the safety of vaccines and recommendation for when they should be given dropped to nearly half, compared to 85% in 2008.

Among the polling results:

70% of Americans agree basic scientific research that advances the frontiers of knowledge, even if it brings no immediate benefits, is necessary and should be supported by the federal government.

80% of Americans say it’s important that elected officials at all levels listen to advice from scientists.

78% of Americans say it’s important that our nation supports research that focuses on improving how our health care system is functioning.

A plurality (44%) say they’re willing to pay more in taxes if they were certain that all of the money would be spent on additional medical research, and

More than half (53%) say it’s important to make the R&D tax credit permanent

56% of Americans favor expanding federal funding for research using embryonic stem cells.

More than half (55%) of Americans are willing to share their personal health information to advance medical research. An even higher percentage (60%) say they will share personal health information so that health care providers can improve patient care, and 46% percent are willing to share information so public health officials can better track disease and disability and their causes.

73% of Americans say the federal government should assign a higher priority to improving education focused on science, technology, engineering and mathematics (STEM) and careers in those fields.

Studies show that certain health problems such as diabetes, heart disease and infant mortality happen more often among minorities or citizens with lower incomes. More than two-thirds of Americans (69%) say it is important to conduct medical or health research to understand and eliminate these differences.

Research!America notes that their online polls are conducted with a sample size of approximately 1,000 U.S. adults, age 18+, with a maximum theoretical sampling error of +/- 3.2%. Data are demographically representative of adult U.S. residents. Polling in this publication was conducted by Zogby Analytics.

February 26, 2015

Celltrion will have to wait longer than expected for FDA's Advisory Committee to review its application for Remsima, a biosimilar for Johnson and Johnsons’ Remicade (infliximab). Yesterday, FDA announced that they would be postponing the meeting of the Arthritis Advisory Committee scheduled for March 17, 2015 where they were going to review the product.

"The postponement is due to information requests pending with the sponsor of the application. A future meeting date will be announced in the Federal Register," states the Agency.

The Advisory Committee was set to review nine proposed indications for Remsima, including treatment of symptoms for Crohn's disease, rheumatoid arthritis, and plaque psoriasis.

Celltrion’s product, being a monoclonal antibody, is more complex than the Sandoz filgrastim biosimilar for which FDA advisers gave a positive recommendation for all five indications in January of this year. The additional data required to show biosimilarity to a monoclonal antibody perhaps may be a reason for the hold-up.

Celltrion has already obtained approval for Remsima from over 50 countries worldwide, including Europe, Canada and Japan.

Just yesterday, Celltrion announced the launch of Remsima in 12 European markets through a partnership with Hospira. Those countries include Austria, Belgium, Denmark, France, Germany, Greece, Italy, Luxembourg, the Netherlands, Spain, Sweden and the UK.

An interesting claim against the pharmaceutical industry (though usually made by only industry's most hardened critics) is that companies don’t want to find a cure—they’d prefer lifelong patients. Recently, Gilead Sciences indeed found a cure for hepatitis C--one that both gets rid of the hepatitis C virus in a patient’s body and does so without many of the terrible side effects that plagued previous therapies. Rather than embracing the medical breakthrough, however, many articles focused on the $84,000 price tag for the full round of treatment. Few mentioned the long-term cost savings now that newly cured patients could lead productive lives without frequent, expensive hospital visits, or would avoid a liver transplant down the road, which might cost a quarter of a million dollars.

Highlighting the focus on cost, California lawmakers have just introduced a bill that would require each manufacturer of a prescription drug that costs "$10,000 or more annually or per course of treatment" to file a yearly report outlining the costs for every such drug.

The report would have to include the following costs paid by the manufacturer and, separately, the costs paid by "any predecessor in the development of the drug":

Total costs for the production of the drug, including the total R&D costs

Total costs of clinical trials and other regulatory costs

Total costs for materials, manufacturing, and administration attributable to the drug

Total costs paid by any entity other than the manufacturer or predecessor for research and development, including any amount from federal, state, or other governmental programs, or any form of subsidies, grants, or other support.

Any other costs to acquire the drug, including costs for the purchase of patents, licensing, or acquisition of any corporate entity owning any rights to the drug while in development.

The total marketing and advertising costs for the promotion of the drug directly to consumers, including, but not limited to, costs associated with direct to consumer coupons and amount redeemed, total marketing and advertising costs for promotion of the drug directly or indirectly to prescribers, and any other advertising for the drug

Manufacturers of, as the bill says, "ultra-high-priced pharmaceuticals" would also have to report an annual history of average wholesale price (AWP) and wholesale acquisition cost (WAC) increases for the drug, including the months each increase took effect, as well as the total profit attributable to the drug, in total dollars and a percentage of total company profits.

California Assembly member David Chiu (D- San Francisco), who introduced the bill, released a Press Release on the proposal. He also provided the following quote to Wall Street Journal: “What we’ve heard for years,” says Chiu, “is that drug pricing is commensurate with costs and that very may well be the case. But we’ve had no transparency about the cost between R&D and the prices charged. Hopefully, this will reduce prices in the long run. But just having the conversation is important. But if your costs are related to your prices, provide the information to help us understand that.”

New Staggered Payment Model

An interesting Forbes article entitled "How Obamacare Pits Insurers Against the Medical Industry," highlights how drug prices will increasingly become a "political event." The writer, Scott Gottlieb, also notes that despite insurance company "surprise" by drug prices, such as Gilead's hep C treatment, Sovaldi, payers knew the likely price up to two years beforehand.

A fundamental issue with insurance company's (and the public's) knee-jerk reaction to a high-cost cure is that they are in essence ignoring that a product's lifetime savings could be far greater than the one-time payment. A recent Reuters article, written by Deena Beasley, outlines an innovative payment model that would help change this dynamic and reward drugmakers for the long-term performance of the medications.

Under the potential model, the cost of an expensive therapy would be amortized over time and contingent on proof of the medication's safety and efficacy. Partial payment at the start of treatment would alleviate what would be a very high cost burden, in favor of spreading out the cost.

The article focuses on firms that are developing gene therapies, which Beasley explains “aim to cure inherited diseases like hemophilia by ‘fixing’ the single faulty gene responsible for the disorder.” Companies in this space include BioMarin Pharmaceutical Inc. in San Rafael, California, and Sangamo BioSciences Inc. in Richmond, California, she notes.

“If these new hemophilia drugs and others like them succeed, a one-time infusion could replace the need for frequent, life-long injections of blood clotting proteins that can cost up to $300,000 a year for a single patient,” Beasley states. Existing hemophilia treatments, “including Pfizer Inc's Xyntha and Baxter International's Advate, are expected to command annual sales of more than $11 billion by 2016.”

The need for a payment model for gene therapies is a ways off stateside, though. "No gene therapies have been approved in the United States, but Europe approved its first gene therapy last year," notes the article. "Glybera treats a rare disorder that clogs the blood with fat and has been cleared for reimbursement in Germany at a price of 850,000 euros, or around $1 million." Joern Aldag, CEO at Dutch biotech firm UniQure NV, which developed Glybera states that Glybera "will be sold for a one-time payment because it is too difficult to measure how well it works."

Uncertainty regarding a treatment's efficacy is one potential issue standing in the way of this new payment model, although the article notes that "[t]he annuity-like payments would be stopped if medical testing, such as the level of clotting protein measured in a patient's blood sample, showed that the therapy was not working."

Beasley also states another consideration in implementing such a model: "Since Americans often switch health insurers, contracts - or even legislation - would be needed to require payers to pick up the ongoing tab for patients who change their coverage."

Despite some clear barriers, Beasley notes that "the interest in new payment models reflects the healthcare industry's intention to find new ways to bolster profits as insurers push back against drug prices." Furthermore, "[s]ome backers of the new model say the payment streams could eventually be packaged and sold to investors, as happens now with securities backed by financial assets like credit card receivables."